2021 ISPE Annual Meeting & Expo: Opening Plenary Focuses on Agility, Collaboration, Innovation
The theme of the 2021 Annual Meeting & Expo is agility, collaboration, and innovation, and these were demonstrated from a number of perspectives in the keynote presentations during the Opening Plenary session on Monday 1 November. This year’s Annual Meeting is being held both in person in Boston, Massachusetts, and virtually, with a total attendance of more than 1,700 people. With participants from 22 countries, this year’s conference is truly a global experience.
The Opening Plenary keynotes provided perspectives from industry and regulatory authorities that focused on the industry’s adaptation to the pandemic. As always, the final presentation represented the patient experience. These highlights from the presentations illustrate the lessons learned over the last 18 months and how changes are shaping the future of the pharmaceutical industry.
Participants were welcomed by Joanne R. Barrick, RPh, Advisor, Global Validation, Eli Lilly and Company and the 2020–2021 ISPE International Board Chair, and Thomas B. Hartman, President and CEO of ISPE. Hartman noted the significance of having both in person and virtual attendees. “Your collective participation in this event makes our collective experience far more meaningful,” Hartman said. He thanked the presenters and speakers both on site and virtual for their participation, and also thanked the Conference Planning Committee, sponsors, and exhibitors for their support.
Pandemic Becomes Opportunity
Robin Kumoluyi, Vice President and Chief Quality Officer, Janssen Pharmaceuticals, Johnson & Johnson Corporation, presented on “Leading with Agility: How the COVID-19 Pandemic was a Catalyst for New Collaboration and Innovation at Johnson & Johnson.”
Kumoluyi became Chief Quality Officer in 2019, just months before the start of the pandemic. While she had envisioned a very different first year in the role, she quickly shifted her focus in the quest to help the company quickly adapt to the realities of operating during the pandemic and making changes to ensure that the company continued to provide products for patients throughout their lifetimes.
She gave some background about the company, tracing the broad scope of the organization, which operates in diverse segments, but noted that there were always questions about whether there were other ways of doing things. The pandemic brought the opportunity to learn about other ways, she said.
As part of her new role, Kumoluyi identified many strengths to the company, including the strong emphasis on quality and compliance, and a focus on product, patient, and continuous improvement. However, Janssen and J&J was focused on traditional thinking and was not heavy on innovation and digital areas that would be needed in the future, she explained. Research and development avoided risk at launch and avoided at-risk capital, and the supply chain was built on traditional and conservative methods and approaches. This led her to wonder if the company was being bold enough, and sufficiently focused on future needs. She wondered about “flipping the ratio” by spending less time being reactive and more time being proactive.
The start of the pandemic brought changes to Kumoluyi’s work and personal life as it did throughout the industry, including changes to her plans for face to face engagement, moving to remote work, and considering work/life for the team. These considerations included how to continue daily work Iabs, how to execute reviews and releases, etc.
Being agile became “more than a buzzword,” since through agility and innovation is how things are made to happen, she said. The agility required during the pandemic also offered the opportunity to “flip the ratio” and become more proactive. In addition to the “typical” way companies adapted to the pandemic, such as online meetings, modified work times and cleaning, dusting off business continuity plans, other changes were happening at Janssen and J&J as well. Thinking began to change about what could be done with IT systems and tools and automation, and this began to alter existing strategies and road maps. Instead of easing into new concepts, which was the way things had been done, the pandemic gave “a platform to pause and think differently, to rip off the Band-Aid and cast aside some conservative thinking.” The only choice the company had was to change its thinking, or risk its ability to care for patients.
From this need came the opportunity to create change through a transformational roadmap that focuses on continuous manufacturing and real-time release, clinical trial innovations, data and digital focus, and the next-gen quality system.
The company began by assessing lessons learned from pandemic changes and identifying new ways of working to carry forward. It looked for examples of innovation: for instance, using a medical device development splitter to address ventilator shortages, or pivoting manufacturing sites to produce hand sanitizer. These were out of norm, but done with speed and agility, she noted. The push to try new things especially in automation were seen as a way to give the strategy and overall vision “life in an accelerated way,” flipping the ratio while keeping the foundation strong and enabling the future and advanced therapies in the pipeline.
This commitment required making sure the entire organization understood the quality strategy and how it would look in the future, Kumoluyi said, telling the story of what the company would look like when the vision was achieved and shared across the organization. The message emphasized the importance of Janssen and J&J’s people, the focus on quality to be maintained, and the key characteristics that will be critical to moving ahead to achieve the strategy.
Being forced to embrace new ways because of the pandemic and the taking away of some conservatism and fear of the unknown has helped move forward, she said. To create the roadmap for making the move forward a reality, projects underway or planned were assessed, since not all would be part of the transformation story. And it was clear that the roadmap would constantly evolve.
One example is the next-gen user experience accelerated focus, which must help navigating today’s siloed workflows. Updated IT tools and processes can streamline and make data more available and allow data needs to be aligned and the next gen program is an opportunity to rethink process and technology together. Robotic process automation and digital adaption tools will help, and full data and analytics strategy is being adopted.
This will be costly but necessary, and she noted that this would not have been considered before the pandemic. The pandemic has become a catalyst to allow the company to move some bold ideas forward.
Although the pandemic has been difficult, it has provided opportunity to accelerate innovation ideas and solutions that may not have been given traction in the past, Kumoluyi said. Being forced to think and work differently has helped overcome some fear of the unknown, and it is important to solidify new ways of working and “capture the moments of genius that came from diversity of thought” during the pandemic.
Innovation and Growth
Midsize pharma companies have also been able to grow and provide more help to patients through the pandemic, as illustrated by “Next Innovation in Pharmaceutical Manufacturing: Creating Growth from Within” from Lou W. Kennedy, CEO and Owner of Nephron Pharmaceuticals, and a member of the ISPE International Board of Directors.
Kennedy is no stranger to innovation: initially a producer of generic respiratory medications, Nephron has grown to almost 2,000 employees and added contract manufacturing and more recently clinical laboratory improvement amendments (CLIA) certification for COVID-19 testing. The company is expanding facilities including a new nitrile glove plant and a vaccine fill-finish facility. More growth is on the way and helping to domestically assist in pandemic-related initiatives is just one of the ways that the company is growing its commitment to serving patients.
Nephron also has a history of innovation in building its employee pool with workforce development programs including internships and externships, apprenticeships, university partnerships, and part-time teacher opportunities.
Regulatory Resilience
Flexibility and agility have also become more the norm for regulatory agencies around the world because of the pandemic. In the presentation “Regulatory Resilience in Challenging Times,” Elizabeth Miller, PharmD, Assistant Commissioner for Medical Products and Tobacco Operations, Office of Regulatory Affairs (ORA), US FDA, described some of the impacts.
She noted that it is “truly remarkable” what has been accomplished since the start of the pandemic. ORA’s adaptation to the circumstances including continuing with inspections despite travel restrictions, and issuing industry guidance; it also continued with mutual reliance efforts and expanded those with Europe and the United Kingdom.
As other speakers in the Opening Plenary session observed, the pandemic has also provided ORA with opportunities to reassess how it works. Mission-ritical inspections were able to continue, primarily in the US although some were done in foreign countries too, she said. An early learning: multiple tools and data sources can be used to conduct inspections, and innovation can be used to develop new tools. Miller said that the pharmaceutical industry including ISPE has been extremely receptive and helpful to ensure that new ideas for oversight could be implemented and accepted.
Miller observed the importance of regulatory agility to maintain resilience and achieve the mission of regulators. This has required more adaptability. The use of data, prioritizing inspections, and using COVID-19 data to assess the number of cases in areas were just some changes. The agency needed to deal with an unprecedented number of emergency use authorizations (EUA); field guidance for new or modified facilities was another facet that needed to be addressed. The FDA released a “Resiliency Roadmap for FDA Inspectional Oversight.”
She noted that over 820 mission-critical inspections were conducted between March 2020 and March 2021. The prioritization approach for inspections will remain in use for the remainder of the pandemic, Miller said, and she said some iteration of that approach will continue later on to complete assignments with the most pressing public health needs.
FDA 704(a)(4) authority to request records reports in lieu of inspections was used broadly during pandemic for preapproval, surveillance, and follow up on some defect reports. Where no explicit authority was in place to request records such as for devices, remote regulatory assessments processes were established to support inspections when the FDA could not be onsite. Firms have had positive feedback to these, she said.
Another tool that was helpful when inspectors could not travel to sites was remote interactive evaluations (RIE); guidance was released in April 2021. RIE can include teleconferences, live streaming, screen sharing records and information, and real-time activities. She cautioned that this is voluntary and not the same as an inspection.
Miller said that the FDA has been evaluating how to optimize operations to use resources as best as possible including oversight of clinical inspections and trials for COVID-19 included. She observed the great collaboration among international regulators during the pandemic months, including work being done on remote assessments and third-country reports. Although terminology may differ, many similar work plans and procedures can be converged.
WHO and the Pandemic
The World Health Organization (WHO) work on getting vaccines and treatments distributed around the world was addressed by Deusdedit Mubangizi, Unit Head, Prequalification Unit (PQT) Regulation & Prequalification Department, World Health Organization, in his presentation “WHO: Agility, Innovation, and Collaboration During the COVID-19 Pandemic.”
Mubangizi cited a statement by Jonas Salk, developer of the first polio vaccine in 1953, that the vaccine belongs to the people. “This is a lesson for us all—that innovations we all make or bring forth are not for us,” he said, “but for the people, all the people.” Working to get the COVID-19 vaccine to all of the world has been a focus for WHO, as well as access to safe, effective, and affordable medical products.
As of the end of September 2021, 6,139M vaccine doses had been administered, and COVAX (the initiative for worldwide distribution of the COVID-19 vaccines) had shipped 307.8M doses to 143 participants. As of that same date, 43% of the population in WHO member states had received at least one vaccine dose. But there were still three countries, territories, and economies that had not yet had immunization programs started, he noted.
Mubangizi applauded the unprecedented levels of agility, innovation, and collaboration that included the work of regulators and the pharmaceutical industry in developing new therapeutics and vaccines to address COVID-19. WHO continues to work with groups around the world including the International Coalition of Medicines Regulatory Authorities (ICMRA), the Regulatory Group, and regional regulatory networks in areas such as in-country approval for use and postapproval monitoring.
Trust is important among industry and regulators in order to share dossiers and information, assessments, and inspection reports, Mubangizi said, and notable in what has been accomplished since the pandemic began.
Innovations such as digital contact tracing and vaccine certificates for international travel, and collaborative efforts among regulatory agencies including open and continuous communication with the industry have been notable.
The establishment of the COVID-19 Technology Access Pool (C-TAP) to realize equitable access to COVID-19 vaccines, diagnostics, and therapeutics through sharing and pooling data, knowledge, and intellectual property is another means of collaboration.
Patient Perspective
Kristen M. Farrell, CI/CT, Vice President, Cure SMA NE Chapter, gave the patient presentation to conclude the Opening Plenary, “Our Journey with SMA: Navigating Uncharted Waters Towards a Port of Calm.”
Farrell’s two children have Spinal Muscular Atrophy (SMA), which she said is often described as “baby Lou Gehrig’s disease.” One in 50 people carry the gene for the neurodegenerative muscular disease, which strikes as one of four types. Type 1 onset occurs before 6 months of age; these patients never sit up. Type 2 onset is at 6–18 months and these patients are able to sit. Farrell’s son Braeden had onset at this age. Type 3 onset occurs after 1 year, so the patients have had the ability to walk. Type 4 onset is after age 30 and normal milestones are reached until onset. SMA patients receive interventional supports and neurology, orthopedics, and physical therapy among other forms of treatment but no pharmaceutical treatments were available until the first FDA approval in 2016.
Farrell and her husband learned about SMA when Braeden was diagnosed at 15 months of age after he was unable to use his legs normally. At the time of Braeden’s diagnosis, the couple were expecting their daughter Kernan. She was tested shortly after birth and also has SMA. Kernan began to show symptoms at 22 months of age.
When Braeden two and a half, the Farrells were approached by their hospital to participate in a clinical trial, which Braeden joined. The result of that trial was Spinraza (nusinersen) from Biogen, which was approved in December 2016 by the FDA, the first pharmaceutical treatment for SMA. Farrell called the drug’s approval “the best Christmas gift of any Christmas.”
Braeden continues in clinical trial today at age 10, and Spinraza has brought “drastic changes and improvement” in motor function, Farrell said. He can stand and walk for some time with braces and a posterior walker for exercise purposes. Obtaining treatment for Kernan came after getting insurance approval, which took some time, but she was able to begin treatments at age 4 years and 3 months, in March 17. She is now almost 9.
Two additional drugs have been developed and approved by the FDA to treat SMA: Zolgensma (onasemnugene abeparvovec-xioi) from Novartis and Evrysdi (risdiplam) from Genentech. Several others are in process, Farrell said. Children can be treated asymptomatically or presymptomatically.
These therapies have been life changing for SMA patients, Farrell said. In the past, untreated SMA patients always needed hip or spine surgery; this is no longer always the case. And now, Farrell said, the SMA community refers to the “waves” that came with the development of the therapies: first wave patients had no treatment; second wave families like the Farrells had a child in clinical trial and early insurance; third wave patients have a choice of multiple treatments and options.
Farrell is active in Cure SMA , the organization originally named Families of SMA when it formed in 1984 by mothers of children with SMA. The organization has grown to include patients, families, medical providers, researchers and more. It focuses on funding research for future breakthroughs, family support, networking and information, legislative advocacy, and hosting conferences and educational opportunities. Cure SMA most recently has been working toward advocating for newborn screening nationwide. Today, 38 states conduct screening, so now 85% of babies in US are screened for SMA, which occurs in 1 in 10,000 births.
Farrell shared photos and videos about the children’s journey and showed them enjoying sports and outdoor activities together as a family. Treatment is now a regular occurrence, with treatments for both children every four months. The pandemic did not have as great an effect on the Farrell family as on other families, since warding off germs and infection were already a regular way of life; SMA patients do not have compromised immune systems but illnesses are harder to manage due to muscles and breathing issues.
“All of this is why your work matters!” Farrell said. “All the agility and collaboration and innovation you put forth every day really trickles down to us. We gain that strength from you and have so much gratitude for that. Thank you all.”
Session Closing
In his closing remarks, Niranjan S. Kulkarni, PhD, Director, Operations Improvement at CRB, and Session Leader and Annual Meeting Committee Chair, thanked the conference planning committee. He noted that this Annual Meeting features over 30 technical sessions with more than 150 presenters, which is an achievement especially during the pandemic environment and its changes. He also thanked the ISPE staff for their assistance in developing the conference.
Disclaimer
This is an abridged, unofficial summary of regulators’ remarks during a presentation at the 2021 ISPE Annual Meeting & Expo that has not been vetted by any agency. The content is an informal and brief synopsis of the presentations, and does not represent official guidance or policy of any agency or regulatory organization.
